Rapid advances in the development of cell and gene therapies over the past several years have heralded “an evolutionary time in medicine.” That’s the opinion of Peter Walters, Director of Advanced Therapies at CRB. That thought reflects data seen in CRB’s annual Horizon Life Sciences report that highlights the rapid expansion of the development of cell and
About 20,000 people in the U.S. live with hemophilia A. It’s a rare X-linked genetic disorder that affects predominantly males and causes their blood to clot poorly when healing wounds. For some, routine daily activities can turn into painful medical emergencies to stop internal bleeding, all because of changes in a single gene that disables
The first tests on eight children show favorable preliminary results, with an adjustment of motor and cognitive skills and improvements in some brain and joint parameters Gene therapy takes steps forward in the fight against rare diseases. Eight children with severe Hurler’s syndrome, a disease associated with reduced life expectancy, benefited from this therapy. The
Chimeric antigen receptor T-cell (CAR T) therapy has revolutionized the field of malignant hematology. CD19-targeted CAR T-cell therapy has resulted in effective, often durable, responses for chemotherapy-refractory B-cell lymphoma and B-acute lymphoblastic leukemia (B-ALL) in prospective clinical trials. CAR T cells against B-cell maturation antigen have also shown overall response rates in the range of
The therapeutic benefit to patients receiving mesenchymal stromal cell (MSC) therapy is not because the injected cells remain viable, but because of cell death, researchers at the Monash Biomedicine Discovery Institute (BDI) have found. In recent years, significant efforts have been made to develop stem cell-based therapies for difficult-to-treat diseases. MSC therapy is regenerative cell-based
The new EFPIA Pipeline Review reveals that infectious, inflammatory, cancer, Alzheimer’s and rare disesases areas are among the top priorities of researchers running clinical trials today to investigate new medicines and vaccines. The comprehensive report found that in 2020 alone, around 5,000 clinical trials were launched – despite the disruption caused by the COVID-19 pandemic. In fact, the volume of trials has
Harnessing the power of cell and gene therapies will require a next-generation level of collaboration not seen before the COVID-19 pandemic. In brief Before cell and gene therapies can meet increasing demand at the necessary speed and scale, the industry must address key challenges. A fractured supply chain, complicated manufacturing and unprecedented pricing are among
In the last issue of Blood, Boulos et al analyze the cost-effectiveness of factor IX gene therapy in patients with severe hemophilia B, in a microsimulation Markov model. Hemophilia is an X-linked disease that affects 400000 people worldwide, of whom 15% have hemophilia B caused by deficiency of factor IX (FIX), which affected the offspring of Queen Victoria,
Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD, Spirovant’s vice president of research, presented the data at the 2021 North American Cystic Fibrosis Conference (NACFC). Her
In a breakthrough for the treatment of aggressive solid cancers, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach. Using the power of large data sets and advanced computational approaches,
